| Abstract: |
In this chapter, we review the current status of allogeneic hematopoietic cell transplants applied to lethal genetic disorders of immunity, including the multiple variants of severe combined immunodeficiency (SCID), Wiskott-Aldrich syndrome and other combined immunodeficiencies, as well as inherited disorders of lymphocyte effector function and regulation. We particularly examine results of HLA-matched related and unrelated marrow grafts, HLA-haploidentical T-cell-depleted parental marrow transplants and cord blood grafts, applied to the treatment of SCID and other combined immunodeficiencies, and evaluate the features of donor and host that foster or limit engraftment, reconstitution of cellular and humoral immunity and long-term disease-free survival. We also assess the contributions of advances in newborn screening, donor selection, conditioning regimens, and supportive care to current outcomes. © 2016 John Wiley & Sons, Ltd. All rights reserved. |
